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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07126262




Registration number
NCT07126262
Ethics application status
Date submitted
29/07/2025
Date registered
17/08/2025
Date last updated
17/08/2025

Titles & IDs
Public title
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
Scientific title
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
Secondary ID [1] 0 0
111-212
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypochondroplasia 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Vosoritide
Treatment: Drugs - Placebo

Experimental: Vosoritide injection with vial and syringe - Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

Placebo comparator: Placebo injection with vial and syringe - Subcutaneous injection of recommended dose of placebo


Treatment: Drugs: Vosoritide
The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

Treatment: Drugs: Placebo
Subcutaneous injection of recommended dose of placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events
Timepoint [1] 0 0
From baseline to end of treatment at 52 weeks
Primary outcome [2] 0 0
Incidence of serious adverse events versus placebo over the course of the study
Timepoint [2] 0 0
From baseline to end of treatment at 52 weeks
Primary outcome [3] 0 0
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Timepoint [3] 0 0
At week 26, at week 52
Primary outcome [4] 0 0
Changes from baseline in heart rate
Timepoint [4] 0 0
At week 13, at week 26, at week 39, at week 52
Primary outcome [5] 0 0
Change from baseline in height Z-score
Timepoint [5] 0 0
At week 52
Primary outcome [6] 0 0
Changes from baseline in respiratory rate
Timepoint [6] 0 0
At week 13, at week 26, at week 39, at week 52
Primary outcome [7] 0 0
Changes from baseline in temperature
Timepoint [7] 0 0
At week 13, at week 26, at week 39, at week 52
Primary outcome [8] 0 0
Changes from baseline in blood pressure
Timepoint [8] 0 0
At week 13, at week 26, at week 39, at week 52
Secondary outcome [1] 0 0
Change in height
Timepoint [1] 0 0
At week 52
Secondary outcome [2] 0 0
Cumulative annualized growth velocity (AGV)
Timepoint [2] 0 0
At week 52
Secondary outcome [3] 0 0
6-month interval AGV
Timepoint [3] 0 0
At week 26, at week 52
Secondary outcome [4] 0 0
Change from baseline in upper to lower body segment ratio
Timepoint [4] 0 0
At week 52
Secondary outcome [5] 0 0
Change from baseline in arm span
Timepoint [5] 0 0
At week 52
Secondary outcome [6] 0 0
Change from baseline in total body (less head) bone mineral density (BMD) Z-score
Timepoint [6] 0 0
At week 52
Secondary outcome [7] 0 0
Change from baseline in lumbar spine BMD Z-score
Timepoint [7] 0 0
At week 52
Secondary outcome [8] 0 0
Change from baseline in total body (less head) bone mineral content (BMC) as measured by DXA
Timepoint [8] 0 0
At week 52
Secondary outcome [9] 0 0
Change from baseline in lumbar spine BMC as measured by DXA
Timepoint [9] 0 0
At week 52
Secondary outcome [10] 0 0
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-8)
Timepoint [10] 0 0
At week 26, at week 52
Secondary outcome [11] 0 0
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Timepoint [11] 0 0
At week 26, at week 52
Secondary outcome [12] 0 0
Elimination half-life of vosoritide (t½)
Timepoint [12] 0 0
At week 26, at week 52
Secondary outcome [13] 0 0
Apparent clearance of vosoritide
Timepoint [13] 0 0
At week 26, at week 52
Secondary outcome [14] 0 0
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Timepoint [14] 0 0
At week 26, at week 52
Secondary outcome [15] 0 0
Time vosoritide is present at maximum concentration (Tmax)
Timepoint [15] 0 0
At week 26, at week 52
Secondary outcome [16] 0 0
Maximum concentration (Cmax) of vosoritide in plasma
Timepoint [16] 0 0
At week 26, at week 52
Secondary outcome [17] 0 0
Change from pre-dose at pre-specified timepoints versus placebo in cyclic guanine monophosphate (cGMP)
Timepoint [17] 0 0
At week 26 and week 52
Secondary outcome [18] 0 0
Incidence of otitis media
Timepoint [18] 0 0
From baseline to end of treatment at 52 weeks
Secondary outcome [19] 0 0
Seizure frequency over the course of the study
Timepoint [19] 0 0
From baseline to end of treatment at 52 weeks

Eligibility
Key inclusion criteria
Key

1. Participants must be 0 to < 36 months of age at randomization.
2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
3. Participants aged 0 to < 12 months must have a height Z-score of = -1.0 SDS andparticipants aged = 12 to < 36 months must have a height Z-score of = -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
4. Participant's weight at the Day 1 visit (pre-treatment) must be = 3 kg.

Key
Minimum age
0 Months
Maximum age
36 Months
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
2. Have an unstable medical condition likely to require surgical intervention during the study period.
3. Taking any of the prohibited medications.
4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
5. Require any investigational agent prior to completion of study period.
6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
9. Have known hypersensitivity to vosoritide or its excipients.
10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/07/2025
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/06/2028
Actual
Sample size
Target
60
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital Melbourne - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
Missouri
Country [8] 0 0
United States of America
State/province [8] 0 0
Wisconsin
Country [9] 0 0
France
State/province [9] 0 0
Paris
Country [10] 0 0
France
State/province [10] 0 0
Rhone-Alps
Country [11] 0 0
Germany
State/province [11] 0 0
North Rhine-Westphalia
Country [12] 0 0
Germany
State/province [12] 0 0
Saarland
Country [13] 0 0
Germany
State/province [13] 0 0
Saxony-Anhalt
Country [14] 0 0
Italy
State/province [14] 0 0
Rome
Country [15] 0 0
Italy
State/province [15] 0 0
Genova
Country [16] 0 0
United Kingdom
State/province [16] 0 0
England

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioMarin Pharmaceutical
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
ICON Clinical Research
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Trial Specialist
Address 0 0
Country 0 0
Phone 0 0
1-800-983-4587
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07126262